The Food and Drug Administration (FDA) has granted Orphan Drug designation to DSP-0390 for the treatment of high-grade glioma.

DSP-0390 is an inhibitor of emopamil-binding protein (EBP), an endoplasmic reticulum membrane protein involved in cholesterol biosynthesis. Increased de novo cholesterol synthesis has been associated with poor survival in patients with glioblastoma multiforme, a form of high-grade glioma.

By inhibiting EBP, DSP-0390 causes an accumulation of zymostenol and zymosterol, which leads to autophagy-mediated cell death in several types of cancer. The cytotoxic activity of DSP-0390 against glioblastoma multiforme, colorectal cancer, and triple-negative breast cancer has been observed in preclinical studies. The product is currently being investigated in a phase 1 trial ( Identifier: NCT05023551) in adults with recurrent, high-grade glioma.

“This designation for DSP-0390 underscores the profound need for novel brain cancer treatment options,” said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. “We are excited to contribute to advancing critical research in brain cancer.”  


Sumitomo Pharma Oncology receives Orphan Drug designation for DSP-0390, an investigational emopamil-binding protein (EBP) inhibitor for the treatment of brain cancer. News release. Sumitomo Pharma Oncology, Inc. Accessed July 18, 2022.